Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

Table 3 Safety information before miglustat initiation and during NPC Registry follow up

Safety information/event	Time period	Patients, N	Patients with events, n (%)**
Seizures	Pre-treatment: present	90	21 (23)
	During follow-up: worsened*	39	13 (33)
	During follow-up: new*	39	13 (33)
Thrombocytopenia	Pre-treatment: present	90	18 (20)
	During follow-up: present*	80	43^† (54)
Tremor	Pre-treatment: present	91	23 (25)
	During follow-up: worsened*	91	6 (7)
	During follow-up: new*	91	2 (2)
Neuropathy	Pre-treatment: present	90	4 (4)
	During follow-up: worsened*	91	7 (8)
	During follow-up: new*	91	1 (1)
Chronic diarrhea^‡	Pre-treatment: present	91	2 (2)
	During follow-up: present*	92	10 (11)
Other^††	During follow-up: present*	92	4 (4)

*Event occurred at least once during follow-up; **percentages calculated according to number of patients with available data; ^†38 mild thrombocytopenia (101–150 × 10⁹/L) and 10 moderate thrombocytopenia (51–100 × 10⁹/L); ^‡diarrhea lasting >3 months; ^††other adverse events comprised: dental cavities, loss of consciousness and circulatory collapse, chronic diarrhea and new seizures (all n = 1)

ISSN: 1750-1172